Traditionally, therapy development progresses from intensive work in target identification to low-success target-specific drug discovery. This project applies an original and unconventional drug-centric rather than target-centric approach, making use of repurposing and lead-like libraries, through a phenotypic screening designed to specifically identify novel small-molecule immunotherapeutics. The results of the SmallDrugRheuma project represent an advancement beyond the state of the art, by identifying two drugs with novel immunomodulatory properties and, as they are repurposing drugs, they have already proved to be safe for their use in humans and, therefore, their way towards a real-world use in clinic is much shorter, thanks to our unbiased, highly efficient, drug-centric approach. Even more, the portfolio of immunomodulatory compounds constitute a huge resource for further research and innovation for the treatment of inflammation and immune-related pathologies.
The work carried out enhance the innovation capacity of the ERA as a whole, as the anti-inflammatory properties of the identified drugs are completely novel and, once protected, will open up a new way for their potential use in RA patients. The results of the project will also step up the development of novel therapies, increasing the potential of citizens to live longer in good health, pushing Europe 2020 objectives. Equally, these results will contribute in the mid-long term towards lessening the impact of RA on EU economy and business, as RA has been recognised by the WHO as a Non-Communicable Disease that can lead to substantial costs for European healthcare.
The results also contribute to Innovation Union and ERA priorities, because addresses the challenge “Health, demographic change and wellbeing” by developing novel drugs for a chronic disease. In particular, these results increase European excellence and benefit the scientific community and society at different levels: (i) Contribution towards science, innovation and generation of knowledge, by providing valuable insights into novel drugs will be useful for specialists in Immunology, but also for those working in a broad range of disciplines and diseases (e.g. cancer); (ii) Contribution to public health and welfare, through the identification of novel drugs, opening the doors to preclinical/clinical trials in chronic diseases and by improving bench-to-bed translation, contributing to the Europe 2020 challenge “Health, demographic change and wellbeing”; (iii) Quick transfer of results to the market through the host’s experience in PPP and IPR planning, bringing rapid benefits to citizens and competitiveness gains.
